Rocket Pharmaceuticals
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2015-01-01
- Employees
- 268
- Market Cap
- $1.7B
- Website
- http://www.rocketpharma.com
- Introduction
Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
- Conditions
- Pyruvate Kinase Deficiency
- Interventions
- Biological: RP-L301
- First Posted Date
- 2019-09-26
- Last Posted Date
- 2024-02-05
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 5
- Registration Number
- NCT04105166
- Locations
- 🇺🇸
Stanford University, Stanford, California, United States
🇪🇸Hospital Infantil Universitario Niño Jesús, Madrid, Spain
🇪🇸Hospital Universitario Fundación Jiménez DÃaz, Madrid, Spain
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
- Conditions
- Fanconi Anemia Complementation Group A
- Interventions
- Biological: RP-L102
- First Posted Date
- 2019-08-28
- Last Posted Date
- 2024-05-22
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 7
- Registration Number
- NCT04069533
- Locations
- 🇪🇸
Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
🇬🇧University College London Great Ormond Street Institute of Child Health (GOSH), London, United Kingdom
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
- Conditions
- Danon Disease
- Interventions
- Biological: RP-A501
- First Posted Date
- 2019-03-20
- Last Posted Date
- 2023-03-03
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 7
- Registration Number
- NCT03882437
- Locations
- 🇺🇸
University of Colorado, Aurora, Colorado, United States
🇺🇸University of California, San Diego, La Jolla, California, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
- Conditions
- Leukocyte Adhesion Defect - Type I
- Interventions
- Biological: RP-L201
- First Posted Date
- 2019-01-31
- Last Posted Date
- 2021-12-07
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Registration Number
- NCT03825783
- Locations
- 🇪🇸
Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
- Conditions
- Fanconi Anemia Complementation Group A
- Interventions
- Biological: RP-L102
- First Posted Date
- 2019-01-24
- Last Posted Date
- 2020-11-24
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 2
- Registration Number
- NCT03814408
- Locations
- 🇺🇸
Stanford University Institute for Stem Cell Biology and Regenerative Medicine Lucille Packard Children's Hospital, Stanford University, Stanford, California, United States
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
- Conditions
- Leukocyte Adhesion Defect - Type I
- Interventions
- Biological: RP-L201
- First Posted Date
- 2019-01-23
- Last Posted Date
- 2023-11-15
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 9
- Registration Number
- NCT03812263
- Locations
- 🇪🇸
Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
🇬🇧University College London Great Ormond Street Institute of Child Health, London, United Kingdom
🇺🇸University of California, Los Angeles, Los Angeles, California, United States